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Clinical Trials /
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Press release /New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gainUpdated results from global…
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Clinical Trials /
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Press release /– A mean increase in CHOP-INTEND of 17.3 was observed at three months post gene therapy in SMA Type 1 U.S. pivotal trial – – All patients in the therapeutic dose cohort in the Phase 1 trial…
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Clinical Trials /
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Press release /SMART study to extend data beyond patient population studied in clinical trials New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world…
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Press release /Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusionAll children (100%) in the presymptomatic intravenous…
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Press release /Results from PROVE-HF trial show significant improvements in measures of cardiac structure and function at six months and one year in heart failure with reduced ejection fraction (HFrEF) patients[1…
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New Novartis data in relapsing MS reinforce benefits of Kesimpta® for first-line and switch patients
Nearly 90% of first-line Kesimpta patients had no disability progression independent of relapse activity (PIRA) for up to six years in an analysis of open-label ALITHIOS extension study1More than 80… -
Press release /– Company to expand study of AVXS-101 into additional SMA populations including pre-symptomatic, older pediatric Type 2 and Type 3 SMA patients – – First patient dosed in Phase 1 trial of…
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